Muscular dystrophy: New drug promises benefit without risk of infection

June 11, 2009

A new drug being studied for the treatment of muscle degenerating diseases has shown promising results. According to a study published today in the British Journal of Pharmacology, Debio 025 is as effective as current drugs but, crucially, does not cause unwanted immunosuppressive effects.

Bethlem Myopathy and Ullrich Congenital Muscular Dystrophy (UCMD) are muscle wasting diseases caused by deficiencies in collagen VI, a component of connective tissue. Patients are usually diagnosed at birth and suffer from muscle weakness that worsens over time. UCMD patients often also suffer respiratory failure, which is complicated by lung infections. Although the drug cyclosporin A (CsA) offers some benefit for these patients, its long term use may be undesirable because it interacts with calcineurin, an important immunoregulatory protein.

"Long-term treatment with CsA is risky because it suppresses the immune system, making patients more susceptible to life-threatening lung infections," says Paolo Bernardi, who led the team at the University of Padova in Italy. "Our findings suggest Debio 025 may provide a safer alternative".

In the study, mice suffering from a muscle-wasting disease similar to human muscular dystrophy were protected when treated with Debio 025. The researchers studied muscle cells from mice given the drug for five days and found decreases in numbers of abnormal muscle fibres similar to those reported in studies of treatment with CsA. They were also able to show that Debio 025, although related to CsA, was not targeting calcineurin.

"This drug has no effects on the immune system and therefore could be used for prolonged periods of time without increasing risk of infection. We should be able to treat children affected by these forms of muscular dystrophy and possibly slow down or even stop the progression of the disease," says Bernardi.

Related Stories

Recommended for you

Short telomeres—the protective caps on the ends of chromosomes—have been previously linked to increased risk of death from heart disease. Now, research by scientists at UC San Francisco and the Veterans Affairs Medical ...

Researchers led by ETH Professor Martin Fussenegger at the Department of Biosystems Science and Engineering (D-BSSE) in Basel have produced artificial beta cells using a straightforward engineering approach.

Medical students are taught that once infected with Toxoplasma gondii—the "cat parasite"—then you're protected from reinfection for the rest of your life. This dogma should be questioned, argue researchers in an Opinion ...

A team of scientists has uncovered details of the cellular mechanisms that control the direct programming of stem cells into motor neurons. The scientists analyzed changes that occur in the cells over the course of the reprogramming ...

Loss of a key protein leads to defects in skeletal development including reduced bone density and a shortening of the fingers and toes—a condition known as brachydactyly. The discovery was made by researchers at Penn State ...

Researchers from the University of Pittsburgh School of Medicine and UPMC have engineered a protein that reverses carbon monoxide (CO) poisoning in mice, a discovery that could potentially lead to the creation of the first ...

0 comments

Please sign in to add a comment.
Registration is free, and takes less than a minute.
Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.